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- Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F
Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F
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Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F: Molecular Therapy
overview : Usher 1F Collaborative News : News & Events : Usher 1F Collaborative
Addgene: pAAV-CMV-N-HA-Pcdh15_mini_V4
Proposed therapy, developed in a Pcdh15-deficient mouse, for progressive loss of vision in human Usher syndrome
Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F
The usherin mutation c.2299delG leads to its mislocalization and disrupts interactions with whirlin and VLGR1
WO2020219990A1 - Aav vectors encoding mini-pcdh15 and uses thereof - Google Patents
The Opportunities and Challenges of Gene Therapy for Treatment of Inherited Forms of Vision and Hearing Loss
Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F
Dual AAV-based PCDH15 gene therapy achieves sustained rescue of visual function in a mouse model of Usher syndrome 1F - ScienceDirect
Finding a window for gene therapy for hereditary deafness
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F: Molecular Therapy
WO2020219990A1 - Aav vectors encoding mini-pcdh15 and uses thereof - Google Patents
Dual AAV-based PCDH15 gene therapy achieves sustained rescue of visual function in a mouse model of Usher syndrome 1F - ScienceDirect
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F: Molecular Therapy
